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Cipaglucosidase alfa, sold under the brand name Pombiliti, and used in combination with miglustat, is a medication used for the treatment of glycogen storage disease type II (Pompe disease). [ 4 ] [ 5 ] Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α ...
The safety and efficacy of Myozyme were assessed in two separate clinical trials in 39 infantile-onset patients with Pompe disease ranging in age from 1 month to 3.5 years at the time of the first infusion. [8] Myozyme treatment prolongs ventilator-free survival and overall survival. Early diagnosis and early treatment lead to much better outcomes.
Miglustat, sold under the brand name Zavesca among others, is a medication used to treat type I Gaucher disease [7] and Pompe disease. [10]It was approved for medical use in the European Union in November 2002, [7] [11] and for medical use in the United States in July 2003.
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Ramona Bates MD, writing for the health news organisation, EmaxHealth, stated that the film brings attention to Pompe disease. [18] Peter Rainer from The Christian Science Monitor mentions that Big Pharma got a surprisingly free pass in the film and that it will come as a surprise to all those sufferers struggling to get orphan drugs developed ...
For the first time in two decades, the Food and Drug Administration (FDA) has approved a new class of medication that provides an alternative to addictive opioids for patients looking to manage ...
William Canfield is a glycobiologist, chief scientific officer and founder of an Oklahoma City-based biotechnology company, Novazyme, which was acquired by Genzyme in August 2001 and developed, among other things, an enzyme that can stabilize (but not cure) Pompe disease, based on Canfield's ongoing research since 1998.
Proton-pump inhibitors have largely superseded the H 2-receptor antagonists, a group of medications with similar effects but a different mode of action, and heavy use of antacids. [3] A potassium-competitive acid blocker (PCAB) revaprazan was marketed in Korea as an alternative to a PPI.
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